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Phase 3 Single-Dose CTX001 in Pediatric Severe Sickle Cell Disease
2024-513978-22-00Phase 3Authorised
- Sponsor
- Vertex Pharmaceuticals
- Start
- Nov 2024
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Cell Therapies
- Countries
- 4
01
Overview
Phase 3, open-label, single-arm trial of exagamglogene autotemcel in sickle cell disease (SCD), sponsored by Vertex Pharmaceuticals. Primary endpoint: VF12 (severe VOC-free for ≥12 consecutive months). Planned start Nov 2024; status: authorised.
02
Drugs
exagamglogene autotemcel
03
Conditions
sickle cell disease (SCD)
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